An Open-label, Single-arm, Post- Authorization Pragmatic Clinical Trial On The Safety And Efficacy Of Xyntha (Moroctocog-alfa (Af-cc), Recombinant Fviii) In Subjects With Hemophilia A In Usual Care Settings In China
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 85
- 试验地点
- 15
- 主要终点
- Percentage of Participants With Factor VIII (FVIII) Inhibitors
研究概览
简要总结
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for approximately 6 months or or approximately 50 exposure days whichever occurs first
详细描述
The purpose of this post-approval study is to provide supplementary information relating to the use of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in Chinese subjects with hemophilia A, especially on the safety and efficacy in different populations of Chinese hemophilia A patients, in particular in pediatric patients <6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FVIII:C <1%).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and/or Female subjects with Hemophilia A.
- •Subjects/parents/legal representatives must be able to comply with registry procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc).
- •Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study.
排除标准
- •Presence of any other bleeding disorder in addition to hemophilia A.
- •Treatment with immunomodulatory therapy (e.g., intravenous immunoglobulin, routine systemic corticosteroids, cyclosporins, anti-TNF agents) within 30 days prior to study entry or planned use for the duration of their study participation.
- •Subjects with a past history of, or current factor VIII inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 BU/mL.
- •Subjects with known hypersensitivity to the active substance or to any of the excipients of Xyntha.
- •Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.
- •Unwilling or unable to follow the terms of the protocol.
- •Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; expectation of poor compliance in provision of observations for study-related documentation), in the opinion of the Investigator.
- •Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation (exception for studies on Xyntha).
- •Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.
研究组 & 干预措施
Intravenous infusions of Xyntha
Enrolled subjects will be treated with intravenous infusions of Xyntha for: • On-Demand treatment, • Surgical Prophylaxis at a dose and frequency prescribed by the subject's treating physician in accordance with the Xyntha label and will be adjusted solely according to medical and therapeutic necessity.
干预措施: Intravenous infusions of Xyntha (Drug)
结局指标
主要结局
Percentage of Participants With Factor VIII (FVIII) Inhibitors
时间窗: From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 exposure days [EDs] whichever occurred first).
Percentage of participants with the product medically important event (MIE) (FVIII inhibitor development during the study).
次要结局
- Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment(From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.)
- Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment(On-Demand Group: Day 1 up to 6 months or 50 EDs whichever occurred first. Surgical Prophylaxis Group: Day of surgery to postoperative period. The duration of postoperative period is specified in previous endpoints.)
- Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)(From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first).)
- Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group(From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.)
- Hemostatic Efficacy for Surgical Prophylaxis Treatment(From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery))
- Actual Estimated Blood Loss for Surgical Prophylaxis Treatment(From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery))
- Percentage of Less Than Expected Therapeutic Effect (LETE) in the On-Demand Setting(From Day 1 up to participants had received treatment for 6 months or participants had achieved 50 EDs whichever occurred first.)
- Number of Confirmed LETE in the Low Recovery Setting(From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.)
- Response Assessment of On-Demand Treatment of Bleeds(From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.)
- Number of Participants With Transfusion Requirement for Surgical Prophylaxis Treatment(From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery))